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WO2017077394A3 - Materials and methods for treatment of hemoglobinopathies - Google Patents

Materials and methods for treatment of hemoglobinopathies Download PDF

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Publication number
WO2017077394A3
WO2017077394A3 PCT/IB2016/001750 IB2016001750W WO2017077394A3 WO 2017077394 A3 WO2017077394 A3 WO 2017077394A3 IB 2016001750 W IB2016001750 W IB 2016001750W WO 2017077394 A3 WO2017077394 A3 WO 2017077394A3
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WIPO (PCT)
Prior art keywords
methods
hemoglobinopathies
cells
materials
hbf
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/IB2016/001750
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French (fr)
Other versions
WO2017077394A2 (en
Inventor
Matthew Hebden PORTEUS
Melanie Ruth Allen
Chad Albert COWAN
Ante Sven LUNDBERG
Michelle I-ching LIN
Jeffrey Li
Thao Thi NGUYEN
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
CRISPR Therapeutics AG
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CRISPR Therapeutics AG
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Filing date
Publication date
Application filed by CRISPR Therapeutics AG filed Critical CRISPR Therapeutics AG
Priority to US15/762,700 priority Critical patent/US12043843B2/en
Priority to ES16829306T priority patent/ES2942309T3/en
Priority to EP16829306.6A priority patent/EP3371306B8/en
Publication of WO2017077394A2 publication Critical patent/WO2017077394A2/en
Publication of WO2017077394A3 publication Critical patent/WO2017077394A3/en
Anticipated expiration legal-status Critical
Priority to US18/752,371 priority patent/US20250171800A1/en
Ceased legal-status Critical Current

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
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    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.
PCT/IB2016/001750 2015-11-04 2016-11-04 Materials and methods for treatment of hemoglobinopathies Ceased WO2017077394A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
US15/762,700 US12043843B2 (en) 2015-11-04 2016-11-04 Materials and methods for treatment of hemoglobinopathies
ES16829306T ES2942309T3 (en) 2015-11-04 2016-11-04 Materials and methods for the treatment of hemoglobinopathies
EP16829306.6A EP3371306B8 (en) 2015-11-04 2016-11-04 Materials and methods for treatment of hemoglobinopathies
US18/752,371 US20250171800A1 (en) 2015-11-04 2024-06-24 Materials and Methods for Treatment of Hemoglobinopathies

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562250823P 2015-11-04 2015-11-04
US62/250,823 2015-11-04
US201662328203P 2016-04-27 2016-04-27
US62/328,203 2016-04-27

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US15/762,700 A-371-Of-International US12043843B2 (en) 2015-11-04 2016-11-04 Materials and methods for treatment of hemoglobinopathies
US18/752,371 Division US20250171800A1 (en) 2015-11-04 2024-06-24 Materials and Methods for Treatment of Hemoglobinopathies

Publications (2)

Publication Number Publication Date
WO2017077394A2 WO2017077394A2 (en) 2017-05-11
WO2017077394A3 true WO2017077394A3 (en) 2017-06-15

Family

ID=57868278

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2016/001750 Ceased WO2017077394A2 (en) 2015-11-04 2016-11-04 Materials and methods for treatment of hemoglobinopathies

Country Status (4)

Country Link
US (2) US12043843B2 (en)
EP (1) EP3371306B8 (en)
ES (1) ES2942309T3 (en)
WO (1) WO2017077394A2 (en)

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US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
AU2016225178B2 (en) 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
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WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
WO2017077394A2 (en) 2015-11-04 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
CN110050074A (en) * 2016-10-05 2019-07-23 新西兰皇家环境科学研究院 RNA-seq for body fluid identification
TW201839136A (en) 2017-02-06 2018-11-01 瑞士商諾華公司 Composition and method for treating hemochromatosis
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US20200140896A1 (en) 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
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US20210180091A1 (en) * 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
BR112020011255A2 (en) 2017-12-05 2020-11-24 Vertex Pharmaceuticals Incorporated human cd34 + hematopoietic stem cells and progenitors modified by crispr-cas9 and uses thereof
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